Update 16th December 2020
The Final Guidance has now been published on the NICE website: www.nice.org.uk/guidance/TA667
TTPNetwork’s response to the NICE Decision on the use of Caplacizumab in the treatment of TTP Nice have today published the following decision:
Caplacizumab with plasma exchange and immunosuppression is recommended, within its marketing authorisation, as an option for treating an acute episode of acquired thrombotic thrombocytopenic purpura (TTP) in adults, and in young people aged 12 years and over who weigh at least 40 kg. Treatment should be started and supervised by physicians experienced in managing thrombotic microangiopathies. It is recommended only if the company provides caplacizumab according to the commercial arrangement.
We are delighted at this decision which comes after more than 18 months of tireless work to ensure that the TTP patient voice was heard during the appraisal process. This news means that every TTP patient in England and Wales* should have access to a treatment, which essentially quickens the time platelet counts returns to normal, reducing the risk of blood clots circulating the body.
Our thanks go to all those patients who agreed to take part in the trials for this drug at a time when their lives hung in the balance. Also, to the teams of doctors and nurses who cared for them.
TTPNetwork would like to pay tribute to every patient and their families who engaged in our surveys and consultations, the results of which enabled us to make a substantial and thorough submission in the form of a report to the NICE Committee. That submission allowed NICE to fully understand the TTP patient journey from diagnosis to remission. It also gave them valuable insight into life living with or caring for a loved one with TTP.
Thank you to the scientists, staff and management team at Ablynx for their many years of research and without whom this treatment would never have been possible. Not forgetting the resident Llamas who played such an important role in the development of Caplacizumab.
Finally, we wish to express our gratitude to Sanofi who have worked hard to bring this treatment to market, and who have enabled many patients to benefit from it under the Compassionate Use scheme whilst the appraisal process was taking place.
For more information about Caplacizumab visit https://www.cablivi.com/what-is-cablivi
For more information about TTPNetwork visit www.ttpnetwork.org.uk
*In September the Scottish Medicines Consortium (SMC) recommended Caplacizumab for use in Scotland. There is no NICE/SMC equivalent Northern Ireland, however the Department of Health, Social Services and Public Safety (DHSSPS) in Northern Ireland link to NICE. This means that they look at any guidance issued by NICE and decide if it is relevant for Northern Ireland.